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European Journal of Human Genetics : EJHG
|
February 11, 2016
Improving the informed consent process in international collaborative rare disease research: effective consent for effective research
Sabina Gainotti, Cathy Turner, Simon Woods, et al.
Molecular Genetics and Metabolism Reports
|
July 22, 2024
Efficacy of avalglucosidase alfa on forced vital capacity percent predicted in treatment-naïve patients with late-onset Pompe disease: A pooled analysis of clinical trials
Tahseen Mozaffar, Lionel Riou França, Jérôme Msihid, et al.
Neuromuscular Disorders : NMD
|
December 4, 2019
Functional characterization of GYG1 variants in two patients with myopathy and glycogenin-1 deficiency
Carola Hedberg-Oldfors, Willem De Ridder, Ognian Kalev, et al.
Neuromuscular Disorders : NMD
|
October 16, 2012
Presymptomatic late-onset Pompe disease identified by the dried blood spot test
Matias Wagner, Amina Chaouch, Juliane S Müller, et al.
Neuromuscular Disorders : NMD
|
July 2, 2021
Late onset Sandhoff disease presenting with lower motor neuron disease and stuttering
Jorge Alonso-Pérez, Ana Casasús, Álvaro Gimenez-Muñoz, et al.
Cardiovascular Research
|
February 17, 2009
Prevention of cardiomyopathy in delta-sarcoglycan knockout mice after systemic transfer of targeted adeno-associated viral vectors
Caroline Goehringer, Désirée Rutschow, Ralf Bauer, et al.
Brain Imaging and Behavior
|
January 3, 2021
Resting-state functional MRI shows altered default-mode network functional connectivity in Duchenne muscular dystrophy patients
Nathalie Doorenweerd, Mischa de Rover, Chiara Marini-Bettolo, et al.
Human Mutation
|
May 24, 2022
Three novel FHL1 variants cause a mild phenotype of Emery-Dreifuss muscular dystrophy
Josefine D S Borch, Thomas Krag, Sonja D Holm-Yildiz, et al.
Neuromuscular Disorders : NMD
|
January 22, 2026
Motor unit magnetic resonance imaging (MUMRI) as a novel biomarker of muscle activity in spinal muscular atrophy
Matthew G Birkbeck, Ian S Schofield, Ian Wilson, et al.
Brain : a Journal of Neurology
|
December 7, 2007
Analysis of the UK diagnostic strategy for limb girdle muscular dystrophy 2A
Emma J Groen, Richard Charlton, Rita Barresi, et al.
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of 38
Search research articles
Search
Showing results (91-100 of 374) with videos related to
Sort By:
Page
of 38
European Journal of Human Genetics : EJHG
|
February 11, 2016
Improving the informed consent process in international collaborative rare disease research: effective consent for effective research
Sabina Gainotti, Cathy Turner, Simon Woods, et al.
Molecular Genetics and Metabolism Reports
|
July 22, 2024
Efficacy of avalglucosidase alfa on forced vital capacity percent predicted in treatment-naïve patients with late-onset Pompe disease: A pooled analysis of clinical trials
Tahseen Mozaffar, Lionel Riou França, Jérôme Msihid, et al.
Neuromuscular Disorders : NMD
|
December 4, 2019
Functional characterization of GYG1 variants in two patients with myopathy and glycogenin-1 deficiency
Carola Hedberg-Oldfors, Willem De Ridder, Ognian Kalev, et al.
Neuromuscular Disorders : NMD
|
October 16, 2012
Presymptomatic late-onset Pompe disease identified by the dried blood spot test
Matias Wagner, Amina Chaouch, Juliane S Müller, et al.
Neuromuscular Disorders : NMD
|
July 2, 2021
Late onset Sandhoff disease presenting with lower motor neuron disease and stuttering
Jorge Alonso-Pérez, Ana Casasús, Álvaro Gimenez-Muñoz, et al.
Cardiovascular Research
|
February 17, 2009
Prevention of cardiomyopathy in delta-sarcoglycan knockout mice after systemic transfer of targeted adeno-associated viral vectors
Caroline Goehringer, Désirée Rutschow, Ralf Bauer, et al.
Brain Imaging and Behavior
|
January 3, 2021
Resting-state functional MRI shows altered default-mode network functional connectivity in Duchenne muscular dystrophy patients
Nathalie Doorenweerd, Mischa de Rover, Chiara Marini-Bettolo, et al.
Human Mutation
|
May 24, 2022
Three novel FHL1 variants cause a mild phenotype of Emery-Dreifuss muscular dystrophy
Josefine D S Borch, Thomas Krag, Sonja D Holm-Yildiz, et al.
Neuromuscular Disorders : NMD
|
January 22, 2026
Motor unit magnetic resonance imaging (MUMRI) as a novel biomarker of muscle activity in spinal muscular atrophy
Matthew G Birkbeck, Ian S Schofield, Ian Wilson, et al.
Brain : a Journal of Neurology
|
December 7, 2007
Analysis of the UK diagnostic strategy for limb girdle muscular dystrophy 2A
Emma J Groen, Richard Charlton, Rita Barresi, et al.
Page
of 38